A record crowd of 22,366 inundated Beantown in early May to attend the 2007 BIO International Convention. They were there to learn about the new and most pressing issues in biotechnologymany of which will impact the practice of pharmacy. Hot topics included the use of biomarkers, personalized medicine, and biosimilars.
Now arguably as famous for his advocacy role in Parkinson's disease as his work in movies and television, keynote speaker Michael J. Fox made the point that academia is more interested in producing papers that are "scientifically interesting" than "medically useful." He urged biotech companies to continue to innovate and accelerate the translation of basic science into improved therapies that can be utilized by patients. Fox's Foundation for Parkinson's Research is currently spending part of its endowment funding biomarker research.
According to Octavi Quintana-Trias, director of health research at the European Commission, a biomarker is an indicator that highlights the clinical stage of a patient. "Biomarkers are important. They measure a patient's clinical status so we can see how the disease progresses or regresses. They make the measurement more objective and much easier," he said in an interview at BIO. But what has been missing from the discovery and development of biomarkers so far has been a systematic method of identifying the proteins and making sense of when and how to use them.
"Biomarkers are not a new idea, but they have been developed over the years in a sort of ad-hoc fashion," explained Barbara Mittleman, M.D., director of the program on public/private partnerships at the National Institutes of Health's Office of Science Policy/Office of Science Policy Analysis. Now, said Mittleman, the NIH is working together in a new consortium with the Food & Drug Administration, the Centers for Medicare & Medicaid Services, the pharmaceutical industry, professional societies, and patient advocacy groupswith the goal not only of identifying and discovering new biomarkers but also of injecting them into the regulatory process in a way that would make sense for drug discovery as well as patient care.
"We're looking for biomarkers for risk, diagnosis, staging disease, response to therapy, and for subgrouping patients into good or bad prognostic groups. We're open to projects looking at any and all of these," she said of the NIH program.
Of course, with advances in biotechnology also comes cost. One session at BIO asked the question, "Can we afford personalized medicine?" The answer is complicated, according to Matthew Hudes, U.S. managing principal of biotechnology at Deloitte Consulting, LLP. "We're in a situation where the science is getting ahead of the business that will allow the new therapies to get to patients," he said. "We don't see enough attention being paid to overcoming the business challenges associated with the smaller populations affected by personalized medicine."
One thing that's wrong with the health economics now, Hudes went on to explain, is that the drug industry is very focused on the blockbuster. Secondly, the payer community is oriented to very short-term relationships with patients. These days, patients typically stay on an insurance plan for a year or two before switching, and some of the benefits of targeted therapies and personalized medicine last over a much longer time period, he pointed out. The other big issue is the overall rising cost of healthcare, and the potential limits that may apply as we begin to spend beyond 16% of the nation's gross domestic product. Hudes believes that personalized medicine can cut that number back not only by improving the effectiveness of a drug but also by lowering the cost associated with the adverse effects of drugs.
"Everyone thinks of personalized medicine as the 'one pill for one patient model.' That model could be extraordinarily expensive to developa unique drug for one patient," Hudes said. He believes that other models, closer to reality, will show the benefits of personalized medicine and targeted therapies, including sharpening the effectiveness and safety of drugs coming to market. He expects that all the major stakeholders will have to be involved to solve the health/economics equation of personalized medicine. They include patients, physicians, employers, third-party payers, and the government.
As for the government, the FDA is now attempting to sort out the controversy surrounding the regulation of generic biologics, or biosimilars. A breakout session, entitled "Biosimilars: The European Approach," discussed how this issue is being dealt with overseas.
"A regulatory pathway already exists in Europe, and a few companies have taken advantage of this regulation and put products on the market," said Charles-Andre Brouwers, a partner and managing director at Boston Consulting Group. He explained how currently in the United States there is no modus operandi to bring a copy of an existing biotech product through the approval process. "There is debate right now as to whether it should be an identical product or a similar product and what degree of sameness is necessary," he said.
Brouwers explained that the European pathway is based on the notion that a very high degree of closeness can be demonstrated. But you cannot absolutely prove that the product is identical, given the nature of a biologic to be inherently variable due to its complex protein structure and a multi-step manufacturing process that is difficult to control.
The Europeans have been very cautious, according to Brouwers. They've started by establishing general principles and guidelines that are applicable only to a small number of products. "The thing that is very important in the European system, and is under discussion in the United States, is the need not only to prove you have a certain degree of similarity, but also to show through clinical trials that the products have similar effects."
Brouwers added that U.S. regulators have invited the European Commission to present their points of view. Therefore, it is likely that there will be similarities between the two pathways, once finalized. "There is still a question about whether you favor economics, safety, or innovation in industry and how you balance among the three."